Long-term efficacy of AAV5-based gene therapy to treat day blind sheep with achromatopsia

(Mary Ann Liebert, Inc./Genetic Engineering News) A study of a large animal model of achromatopsia caused by a mutation in the CNGA3 gene that was treated with a single injection of CNGA3 gene therapy delivered using an AAV5 vector revealed findings reported long-term follow-up findings that show promise for the efficacy and safety of this therapeutic approach.